Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy | DuchenneXchange

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Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy

key information

study id #: NCT03508947

condition: Duchenne Muscular Dystrophy

status: active, not recruiting

purpose:

This is a Phase 1, double-blind, placebo-controlled, single ascending dose cohort study to evaluate the safety, tolerability, and plasma concentrations of WVE-210201 in ambulatory and non-ambulatory male pediatric patients with DMD amenable to exon 51 skipping intervention.

intervention:
WVE-210201, Placebo

mechanism of action:
Exon-skipping to promote dystrophin production

results:
https://clinicaltrials.gov/ct2/show/results/NCT03508947

study details

start date: January 24, 2018

estimated completion: March 2019

phase of development:
Phase 1

size / enrollment: 40

primary outcomes:

Safety: Number of patients with adverse events (AEs) [Time Frame: Day 1 to Day 85 (end of study)]

Safety: Severity of AEs [Time Frame: Day 1 to Day 85 (end of study)]

Safety: Number of patients with serious AEs (SAEs) [Time Frame: Day 1 to Day 85 (end of study)]

Safety and Tolerability: Number of patients who withdraw due to AEs [Time Frame: Day 1 to Day 85 (end of study)]

secondary outcomes:

Safety: Number of patients with adverse events (AEs) [Time Frame: Day 1 to Day 85 (end of study)]

Safety: Severity of AEs [Time Frame: Day 1 to Day 85 (end of study)]

Safety: Number of patients with serious AEs (SAEs) [Time Frame: Day 1 to Day 85 (end of study)]

Safety and Tolerability: Number of patients who withdraw due to AEs [Time Frame: Day 1 to Day 85 (end of study)]
Pharmacokinetics (PK): Maximum observed concentration (Cmax) [Time Frame: Day 1, Day 2, and Day 8]

PK: Time of occurrence of Cmax (tmax) [Time Frame: Day 1, Day 2, and Day 8]

PK: Area under the plasma concentration-time curve (AUC 0-t) [Time Frame: Day 1, Day 2, and Day 8]

inclusion criteria:
• Diagnosis of Duchenne muscular dystrophy (DMD) based on clinical phenotype with increased serum creatine kinase
• Documented mutation in the Dystrophin gene associated with DMD that is amenable to exon 51 skipping
• Ambulatory or non-ambulatory male patients aged >=5 - <=18 years
• Stable pulmonary and cardiac function as measured by:
Reproducible percent predicted forced vital capacity (FVC) >=50%;
Left ventricular ejection fraction (LVEF) >55% in patients <10 years of age and >45% in patients >=10 years of age, as measured (and documented) by echocardiogram within one year prior to enrollment into the study.

exclusion criteria:
• Severe cardiomyopathy; cardiomyopathy that is managed by angiotensin-converting enzyme (ACE) inhibitors or beta blockers is acceptable provided the patient meets the LVEF inclusion criteria.
• Need for mechanical or non-invasive ventilation OR anticipated need for mechanical or non-invasive ventilation within the next year, in the opinion of the Investigator.
• Changes in nutritional or herbal supplements or concomitant medications within 1 month prior to Screening visit or plans to modify dose or regimen during the study.
• Currently on anticoagulants or antithrombotics.
• Received treatment with eteplirsen or ataluren within the past 14 weeks.
• Received prior treatment with drisapersen.
• Received any investigational drug within the past 3 months or 5 half-lives, whichever is longer.